ARCA biopharma, Inc. is a Colorado-based biopharmaceutical company developing genetically-targeted therapies for cardiovascular diseases.
ARCA was founded on the belief that a personalized medicine approach to drug development, tailoring medical treatment to the individual genetic characteristics of each patient, can enable more effective therapies, improve patient outcomes and reduce healthcare costs. ARCA’s lead development program is intended to be a direct implementation of those ideas.
The Company's lead product candidate, GencaroTM (bucindolol hydrochloride), is an investigational, pharmacologically unique beta-blocker and mild vasodilator being developed for the prevention of atrial fibrillation in patients with heart failure. ARCA has identified common genetic variations in the cardiovascular system that it believes interact with Gencaro’s pharmacology and may predict individual patient response to Gencaro, giving it the potential to be the first genetically-targeted atrial fibrillation prevention treatment.
We are planning to initiate GENETIC-AF, a Phase 2B/3 clinical study of Gencaro in AF patients with heart failure and reduced left ventricular ejection fraction ("HFREF"). We believe AF is an attractive indication for Gencaro because data from BEST, the previously conducted Phase 3 heart failure trial involving Gencaro in 2,708 HF patients, suggest Gencaro may have a potentially significant effect in reducing and/or preventing AF. Based on the BEST trial, we believe Gencaro's prevention of AF in HF patients is pharmacogenetically regulated, similar to the effects on HF clinical endpoints.
GENETIC-AF is planned as a Phase 2B/3, multi-center, randomized, double-blind clinical trial comparing Gencaro to metoprolol CR/XL for prevention of AF in patients with heart failure and reduced left ventricular ejection fraction (HFREF). ARCA plans to enroll only patients with the genetic variant of the beta-1 cardiac receptor which the Company believes responds most favorably to Gencaro. GENETIC-AF has an adaptive design, under which the Company plans to initiate it as a Phase 2B study in approximately 200 patients and then, depending on the results of an interim analysis by the trial Data Safety Monitoring Board (DSMB), expand the trial to a Phase 3 study by enrolling an estimated additional 420 patients. The Company now anticipates that patient enrollment in GENETIC-AF will begin in the first quarter of 2014. The Company is collaborating with Medtronic, Inc. on the Phase 2B portion of GENETIC-AF
Under the collaboration with Medtronic, ARCA plans to conduct a substudy that will include continuous monitoring of the cardiac rhythms of all 200 patients enrolled during the Phase 2B portion of GENETIC-AF. Each patient will have heart rhythm monitoring via a Medtronic device, either a previously implanted cardiac resynchronization or defibrillation device, or a previously or newly inserted Reveal® loop recorder. The collaboration substudy will measure AF burden, defined as a patient’s actual time in AF regardless of symptoms. For the DSMB interim analysis, AF burden and the primary endpoint of the study, time to recurrence of symptomatic AF after electrical cardioversion, or death, will be reviewed by the DSMB to determine if there is sufficient potential for a statistically significant efficacy signal to be determined for all patients enrolled in the Phase 2B/3 study.